Genetic engineering platforms and research translated honestly.
Vertex Pharmaceuticals has presented the first clinical data for Casgevy in children ages 5-11 at the American Society of Hematology annual meeting, on December 6, 2025, in Orlando. All evaluated children in the sickle cell cohort remained free of vaso-occlusive crises for up to two years after…
Epigenetics is the field of study focused on changes in DNA that do not involve alterations to the underlying sequence, per NHGRI's Talking Glossary, which also calls the field epigenomics. The commercial and scientific interest sits in the platforms that read those changes at scale: sequencing-…
Cell and gene therapy manufacturing produces either a viral vector carrying a therapeutic gene or living cells engineered with one, under good manufacturing practice controls. FDA's January 2020 CMC guidance defines the IND baseline: enough information to assure safety, identity, quality,…
Base editing and prime editing are precision genome editing methods that rewrite DNA without the double-strand break standard CRISPR cutting relies on. Base editing, first described in Nature in 2016, converts one DNA base into another; prime editing, described in Nature in 2019, writes new…
Gene-editing therapies are regulated in the United States by FDA's Center for Biologics Evaluation and Research as human gene therapy products, reviewed through the Investigational New Drug pathway under 21 CFR 312.23. The operative document is FDA's January 2024 guidance on editing of somatic…
RNA therapeutics are drugs whose active substance is nucleic acid — small interfering RNA, antisense oligonucleotides, or messenger RNA — acting on the instructions a cell reads rather than blocking a finished protein. The category earned its standing one approval at a time: FDA approved Qfitlia…